Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study
Thrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and life-threatening complication. Thus, new prevention and treatment strategies for post-HSCT thrombocytopenia are urgently required. In recent studies, thrombopoietin receptor agonists (TPO-RA) fo...
| Main Authors: | , , , , , , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Frontiers Media S.A.
2023-02-01
|
| Series: | Frontiers in Pediatrics |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fped.2023.1099372/full |
| _version_ | 1797924461628882944 |
|---|---|
| author | Yongsheng Ruan Wei Cao Tingting Luo Xuan Liu Qiujun Liu Yuhua Xiao Cuiling Wu Danfeng Xie Yuqiong Ren Xuedong Wu Xiaoqin Feng |
| author_facet | Yongsheng Ruan Wei Cao Tingting Luo Xuan Liu Qiujun Liu Yuhua Xiao Cuiling Wu Danfeng Xie Yuqiong Ren Xuedong Wu Xiaoqin Feng |
| author_sort | Yongsheng Ruan |
| collection | DOAJ |
| description | Thrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and life-threatening complication. Thus, new prevention and treatment strategies for post-HSCT thrombocytopenia are urgently required. In recent studies, thrombopoietin receptor agonists (TPO-RA) for treating post-HSCT thrombocytopenia indicated efficiency and safety. The improved effect of post-HSCT thrombocytopenia in adults was found in the administration of avatrombopag which was a new TPO-RA. However, there was no relevant study in the children's cohort. Herein, we retrospectively analyzed the effect of avatrombopag in post-HSCT thrombocytopenia in children. As a result, the overall response rate (ORR) and complete response rate (CRR) were 91% and 78%, respectively. Furthermore, both cumulative ORR and CRR were significantly lower in the poor graft function (PGF)/secondary failure of platelet recovery (SFPR) group compared to the engraftment-promotion group (86.7% vs. 100%, p = 0.002 and 65.0% vs. 100%, p < 0.001, respectively). Achieving OR required a median of 16 days in the PGF/SFPR group while 7 days in the engraftment-promotion group (p = 0.003). Grade III–IV acute graft vs. host disease and inadequate megakaryocytes were identified as risk factors of CRR only in univariate analysis (p = 0.03 and p = 0.01, respectively). No severe adverse events were documented. Conclusively, avatrombopag is an alternatively efficient and safe agent for treating post-HSCT thrombocytopenia in children. |
| first_indexed | 2024-04-10T15:02:49Z |
| format | Article |
| id | doaj.art-2d77fffa9a3044cfb57afdb9ffa50465 |
| institution | Directory Open Access Journal |
| issn | 2296-2360 |
| language | English |
| last_indexed | 2024-04-10T15:02:49Z |
| publishDate | 2023-02-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pediatrics |
| spelling | doaj.art-2d77fffa9a3044cfb57afdb9ffa504652023-02-15T09:22:46ZengFrontiers Media S.A.Frontiers in Pediatrics2296-23602023-02-011110.3389/fped.2023.10993721099372Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot studyYongsheng RuanWei CaoTingting LuoXuan LiuQiujun LiuYuhua XiaoCuiling WuDanfeng XieYuqiong RenXuedong WuXiaoqin FengThrombocytopenia following allogeneic hematopoietic stem cell transplantation (allo-HSCT) is a common and life-threatening complication. Thus, new prevention and treatment strategies for post-HSCT thrombocytopenia are urgently required. In recent studies, thrombopoietin receptor agonists (TPO-RA) for treating post-HSCT thrombocytopenia indicated efficiency and safety. The improved effect of post-HSCT thrombocytopenia in adults was found in the administration of avatrombopag which was a new TPO-RA. However, there was no relevant study in the children's cohort. Herein, we retrospectively analyzed the effect of avatrombopag in post-HSCT thrombocytopenia in children. As a result, the overall response rate (ORR) and complete response rate (CRR) were 91% and 78%, respectively. Furthermore, both cumulative ORR and CRR were significantly lower in the poor graft function (PGF)/secondary failure of platelet recovery (SFPR) group compared to the engraftment-promotion group (86.7% vs. 100%, p = 0.002 and 65.0% vs. 100%, p < 0.001, respectively). Achieving OR required a median of 16 days in the PGF/SFPR group while 7 days in the engraftment-promotion group (p = 0.003). Grade III–IV acute graft vs. host disease and inadequate megakaryocytes were identified as risk factors of CRR only in univariate analysis (p = 0.03 and p = 0.01, respectively). No severe adverse events were documented. Conclusively, avatrombopag is an alternatively efficient and safe agent for treating post-HSCT thrombocytopenia in children.https://www.frontiersin.org/articles/10.3389/fped.2023.1099372/fullavatrombopagthrombocytopeniaallogeneic hematopoietic stem cell transplantationthrombopoietin receptor agonistpoor graft functionsecondary failure of platelet recovery |
| spellingShingle | Yongsheng Ruan Wei Cao Tingting Luo Xuan Liu Qiujun Liu Yuhua Xiao Cuiling Wu Danfeng Xie Yuqiong Ren Xuedong Wu Xiaoqin Feng Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study Frontiers in Pediatrics avatrombopag thrombocytopenia allogeneic hematopoietic stem cell transplantation thrombopoietin receptor agonist poor graft function secondary failure of platelet recovery |
| title | Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study |
| title_full | Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study |
| title_fullStr | Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study |
| title_full_unstemmed | Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study |
| title_short | Avatrombopag for the treatment of thrombocytopenia in children's patients following allogeneic hematopoietic stem-cell transplantation: A pilot study |
| title_sort | avatrombopag for the treatment of thrombocytopenia in children s patients following allogeneic hematopoietic stem cell transplantation a pilot study |
| topic | avatrombopag thrombocytopenia allogeneic hematopoietic stem cell transplantation thrombopoietin receptor agonist poor graft function secondary failure of platelet recovery |
| url | https://www.frontiersin.org/articles/10.3389/fped.2023.1099372/full |
| work_keys_str_mv | AT yongshengruan avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT weicao avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT tingtingluo avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT xuanliu avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT qiujunliu avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT yuhuaxiao avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT cuilingwu avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT danfengxie avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT yuqiongren avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT xuedongwu avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy AT xiaoqinfeng avatrombopagforthetreatmentofthrombocytopeniainchildrenspatientsfollowingallogeneichematopoieticstemcelltransplantationapilotstudy |